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Access through your institution Buy or subscribe In the May 2003 issue of this journal, Dror _et al_1 reported two children with dyskeratosis congenita (DC) who successfully underwent
hematopoietic stem-cell transplantation (HSCT) after low-intensity conditioning. The fludarabine-based conditioning regimen was chosen to avoid the complications described after HSCT for DC,
particularly lethal pulmonary and endothelial problems.2, 3, 4, 5 The patients successfully engrafted without severe early or late side effects. Two other successful transplants after
low-intensity fludarabine-based conditioning regimens have been reported.6, 7 We thus used the regimen described by Dror _et al_ for a 15-year-old boy with DC. Symptoms of DC were esophageal
stenosis with dysphagia, nail dystrophy, reticulated hyperpigmentation, anemia and thrombocytopenia with myelodysplasia. Chromosome breakage studies excluded Fanconi anemia. At the time of
transplantation, the patient already had received 62 packed red cell transfusions and 37 platelet transfusions. Conditioning included fludarabine 30 mg/m2/day i.v. from day −10 to day −5
(total dose 180 mg/m2), cyclophosphamide 60 mg/kg/day i.v. on days −6 and −5 (total dose 120 mg/kg) and equine antithymocyte globulin (ATGAM, Upjohn, Kalamazoo, MI, USA) 40 mg/kg/day from
day −4 to −1 (total dose 160 mg/kg). The patient received a non-T-cell-depleted unrelated bone marrow graft, matched for HLA loci A and B at the serological level, and for the DRB1 locus at
high-resolution molecular level. The cell dose was 3.8 × 108 nucleated cells/kg. For graft-versus-host disease (GVHD) prophylaxis, the patient was planned to receive i.v. cyclosporine twice
daily starting on day −1, targeting a whole blood trough level of 300–400 ng/ml, methotrexate 15 mg/m2 i.v. on day +1 and 10 mg/m2 i.v. on days +3 and +6, and methylprednisolone 0.4
mg/kg/dose twice daily from day –4 to day +28 with subsequent dosage reduction. He received granulocyte-colony-stimulating factor (G-CSF) 5 _μ_g/kg/day s.c. from day +5 until neutrophil
counts were >1.0 × 109/l for at least two consecutive days. He also received intravenous immunoglobulin 0.5 g/kg weekly, trimethoprim-sulfamethoxazole 75 mg trimethoprim/m2 twice daily
orally starting 1 day before conditioning until day –2, fluconazole 5 mg/kg/day orally from day −3 and acyclovir 250 mg/m2 twice daily i.v. from day –3 to +20. Donor serology for
cytomegalovirus was positive, whereas patient serology was negative. This is a preview of subscription content, access via your institution ACCESS OPTIONS Access through your institution
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Contact customer support REFERENCES * Dror Y, Freedman MH, Leaker M et al. Low-intensity hematopoietic stem-cell transplantation across human leucocyte antigen barriers in dyskeratosis
congenita. _Bone Marrow Transplant_ 2003; 31: 847–850. Article CAS Google Scholar * Langston AA, Sanders JE, Deeg HJ et al. Allogeneic marrow transplantation for aplastic anaemia
associated with dyskeratosis congenita. _Br J Haematol_ 1996; 92: 758–765. Article CAS Google Scholar * Rocha V, Devergie A, Socie G et al. Unusual complications after bone marrow
transplantation for dyskeratosis congenita. _Br J Haematol_ 1998; 103: 243–248. Article CAS Google Scholar * Conter V, Johnson FL, Paolucci P et al. Bone marrow transplantation for
aplastic anemia associated with dyskeratosis congenita. _Am J Pediatr Hematol Oncol_ 1988; 10: 99–102. Article Google Scholar * Yabe M, Yabe H, Hattori K et al. Fatal interstitial
pulmonary disease in a patient with dyskeratosis congenita after allogeneic bone marrow transplantation. _Bone Marrow Transplant_ 1997; 19: 389–392. Article CAS Google Scholar * Nobili B,
Rossi G, De Stefano P et al. Successful umbilical cord blood transplantation in a child with dyskeratosis congenita after a fludarabine-based reduced-intensity conditioning regimen. _Br J
Haematol_ 2002; 119: 573–574. Article Google Scholar * Gungor T, Corbacioglu S, Storb R, Seger RA . Nonmyeloablative allogeneic hematopoietic stem cell transplantation for treatment of
dyskeratosis congenita. _Bone Marrow Transplant_ 2003; 31: 407–410. Article CAS Google Scholar * Daly AS, Xenocostas A, Lipton JH . Transplantation-associated thrombotic microangiopathy:
twenty-two years later. _Bone Marrow Transplant_ 2002; 30: 709–715. Article CAS Google Scholar * Lesesve JF, Salignac S, Lecompte T, Bordigoni P . Automated measurement of schistocytes
after bone marrow transplantation. _Bone Marrow Transplant_ 2004; 34: 357–362. Article Google Scholar * Ertault-Daneshpouy M, Leboeuf C, Lemann M et al. Pericapillary hemorrhage as
criterion of severe human digestive graft-versus-host disease. _Blood_ 2004; 103: 4681–4684. Article CAS Google Scholar Download references AUTHOR INFORMATION AUTHORS AND AFFILIATIONS *
Service d'Hématologie-Oncologie, Hopital Sainte-Justine, Montreal, Canada P Brazzola, M Duval, J C Fournet, F Gauvin, J H Dalle, J Champagne & M A Champagne Authors * P Brazzola
View author publications You can also search for this author inPubMed Google Scholar * M Duval View author publications You can also search for this author inPubMed Google Scholar * J C
Fournet View author publications You can also search for this author inPubMed Google Scholar * F Gauvin View author publications You can also search for this author inPubMed Google Scholar *
J H Dalle View author publications You can also search for this author inPubMed Google Scholar * J Champagne View author publications You can also search for this author inPubMed Google
Scholar * M A Champagne View author publications You can also search for this author inPubMed Google Scholar RIGHTS AND PERMISSIONS Reprints and permissions ABOUT THIS ARTICLE CITE THIS
ARTICLE Brazzola, P., Duval, M., Fournet, J. _et al._ Fatal diffuse capillaritis after hematopoietic stem-cell transplantation for dyskeratosis congenita despite low-intensity conditioning
regimen. _Bone Marrow Transplant_ 36, 1103–1105 (2005). https://doi.org/10.1038/sj.bmt.1705171 Download citation * Published: 03 October 2005 * Issue Date: 01 December 2005 * DOI:
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